Patient-reported outcomes in relapsed/refractory multiple myeloma treated with melflufen plus dexamethasone: analyses from the Phase II HORIZON study.
Alessandra LaroccaXavier LeleuCyrille TouzeauJoan BladéAgne PanerMaria-Victoria Mateos-MantecaMichele CavoChristopher MaiselAdrían AlegreAlbert OriolAnastasios RaptisPaula Rodriguez OteroAmitabha MazumderJacob P LaubachOmar NadeemAnna SandbergMarie OrreAnna TorrångNicolaas A BakkerPaul Gerard RichardsonPublished in: British journal of haematology (2021)
Relapsed/refractory multiple myeloma (RRMM) is known to have a high burden of disease and complications associated with refractoriness to prior lines of therapy. Severe pain and fatigue symptoms and impairments in physical and emotional functioning have been strongly linked to reduced health-related quality of life (HRQoL) in patients with RRMM. Assessment of patient reported-outcome measures from the pivotal, Phase II HORIZON study (OP-106; NCT02963493) in patients with RRMM (n = 64) demonstrated that melphalan flufenamide (melflufen) plus dexamethasone treatment preserved HRQoL. Patients had clinically meaningful improvements, even after eight treatment cycles, in relevant scales such as global health status/QoL, physical functioning, emotional functioning, pain, and fatigue. Patients with triple-class-refractory disease (n = 50) displayed similar improvements. Patient-reported outcome deterioration was delayed for a substantial amount of time in patients who experienced a response to melflufen plus dexamethasone treatment relative to patients who did not experience a response. These findings support the notion that treatment with melflufen plus dexamethasone may sustain or improve HRQoL over time in patients with RRMM, including in patients with triple-class-refractory disease for whom outcomes are generally worse. The clinical benefits observed in patients from the HORIZON trial are encouraging and supportive of translation into real-world practice.
Keyphrases
- patient reported outcomes
- phase ii
- multiple myeloma
- clinical trial
- patient reported
- high dose
- end stage renal disease
- low dose
- chronic pain
- ejection fraction
- physical activity
- acute lymphoblastic leukemia
- newly diagnosed
- primary care
- open label
- acute myeloid leukemia
- stem cells
- type diabetes
- mental health
- randomized controlled trial
- insulin resistance
- depressive symptoms
- combination therapy
- metabolic syndrome
- prognostic factors
- mesenchymal stem cells
- study protocol
- cell therapy
- risk factors
- skeletal muscle
- weight loss
- quality improvement
- hodgkin lymphoma